Study on the efficacy of trametinib in the treatment of refractory/recurrent langerhans cell histiocytosis in children

Status

Recruiting

Phases

Phase 4

Study type

Interventional

Source

ChiCTR

Registry ID

ChiCTR2100045125

Enrollment

Unknown

Registered

2021-04-07

Start date

2021-04-07

Completion date

Unknown

Last updated

2021-11-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Langerhans cell histiocytosis

Interventions

Trametinib group:Treatment of trametinib

Eligibility

Sex/Gender

All

Age

No minimum to 18 Years

Inclusion criteria

Inclusion criteria: 1. Patients are less than 18 years of age; 2. Patients are diagnosed as LCH by clinical and pathological examination; 3. The ERBB3, BRAF, ARAF, KRAS, NRAS, MEK (MAP2K1 and MAP2K2) or other upstream gene mutations of MEK is positive in biopsied tissue or plasma before treatment; 4. Refractory/recurrent patients with BRAF mutations after treatment of dabrafenib, or refractory/recurrent patients with MEK or other upstream gene (non-BRAF gene) mutations of MEK after chemotherapy with CCHG-LCH-2019 regimen; or patients are unable to tolerate chemotherapy because of severe side effects of chemotherapy or complicated with hemophagocytic lymphohistiocytosis (HLH); or cell-free mutated DNA do not turn negative during the treatment of chemotherapy or dabrafenib, or return to positive after withdrawal; 5. The function of organs is well; 6. Informed consent is obtained from guardians of the patients and the patients (more than 8 years old).

Exclusion criteria

Exclusion criteria: 1. Subjects who are allergic to the components of Trametinib; 2. Have previously received or participated in clinical studies of other experimental drugs; 3. Organ dysfunction or other uncontrollable diseases; have a history of heart disease such as myocardial infarction, unstable angina pectoris, peripheral vascular disease, familial QT interval prolongation, abnormal morphology of heart valve; history of other malignant tumors such as leukemia, lymphoma, etc; 4. Subjects who are unable to comply during the trial and / or follow-up phase.

Design outcomes

Primary

Measure	Time frame
objective response rate;disease control rate;progression-free survival rate;overall survival rate;	—

Countries

China

Contacts

Public ContactZhang Rui

Beijing Children's Hospital, Capital Medical University

ruizh1973@126.com+86 18611106187

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026